Sarepta Therapeutics Announces that Phase 1/2a Duchenne Muscular Dystrophy (DMD) Micro-Dystrophin Gene Therapy Trial Placed on Clinical Hold Due to an Out-of-Specification Production Lot; No Observed Safety Events
-- Out-of-specification lot resulted from the presence of trace levels of DNA fragment in research-grade raw material plasmid sourced from third-party manufacturer --
-- Fragment fully characterized; preliminary testing and analysis indicates no safety signals --
-- Subject to
-- Clinical timeline to commence dosing of patients in pivotal trial by year-end 2018 remains on track --
-- Sarepta to host conference call on
“Patient safety is our top priority at Sarepta as we know it is for Nationwide Children’s
Sarepta will host a conference call today,
This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding Research Institute’s action plan, including the plan to use GMP-s plasmid for all future production lots; the preliminary in-vivo tests performed by the
These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include, among others: preliminary testing results do not necessarily predict final results; the response to the FDA’s clinical hold letter may take longer than expected; the expected clinical timeline for the micro-dystrophin gene therapy trial may be delayed; we may not be able to execute on our business plans, including meeting our expected or planned regulatory milestones and time-lines, research and clinical development plans, and bringing our product candidates to market, for various reasons including that study data may not consistently or sufficiently demonstrate the safety or efficacy of any of our product candidates, possible limitations of Company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the CHMP on eteplirsen or the United States Patent and Trademark Office with respect to patents that cover our product candidates; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended
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Source: Sarepta Therapeutics, Inc.
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Sarepta IR Presentation (December 2017)